Xiangning Yuan, Tong Li, Yudong Liu, Min Wang, Xiaoxia Zuo, Ying Jiang, Xuan Zhang
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The OR rate was 60.0% (9/15), 66.7% (10/15) and 73.3% (11/15) at week 4, 8 and 12, respectively, in the RTX group. It remained at 72.7% (8/11) during week 4-12 in the BLM group. Excluding the data of three deceased patients, the OR rate dropped at week 24 in both groups (RTX vs BLM, 61.5% (8/13) vs 70.0% (7/10), p=1.000). At week 24, four patients with OR in both groups successfully withdrew GCs to <15 mg prednisone (RTX vs BLM, 40% (4/10) vs 66.7% (4/6), p=0.608). The serum C3 level did not significantly change, whereas the serum immunoglobulin G level significantly decreased at week 4, 8 and 12 in both groups. There were three patients with serious adverse effects who died of severe pneumonia during weeks 12-24.</p><p><strong>Conclusions: </strong>BLM may be a safe and effective alternative to RTX for CTD-ITP refractory to GCs plus ISAs.</p>","PeriodicalId":18126,"journal":{"name":"Lupus Science & Medicine","volume":"12 1","pages":""},"PeriodicalIF":3.7000,"publicationDate":"2025-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease.\",\"authors\":\"Xiangning Yuan, Tong Li, Yudong Liu, Min Wang, Xiaoxia Zuo, Ying Jiang, Xuan Zhang\",\"doi\":\"10.1136/lupus-2025-001501\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Objectives: </strong>Immune thrombocytopenia (ITP) is a haematological manifestation secondary to connective tissue disease (CTD). Many patients with CTD-ITP are refractory to glucocorticoids (GCs) plus immunosuppressant agents (ISAs); rituximab (RTX) is the recommended second-line therapy. Belimumab (BLM) shows efficacy against CTD. We compared the efficacy and safety of RTX and BLM.</p><p><strong>Methods: </strong>Data of patients with CTD-ITP refractory to GCs plus ISAs administered were collected. The data of 11 patients with refractory CTD-ITP who received BLM were compared with those of 15 patients treated with RTX.</p><p><strong>Results: </strong>At week 2, BLM resulted in a better overall response (OR) than RTX (72.7% vs 26.7%, p=0.045). The OR rate was 60.0% (9/15), 66.7% (10/15) and 73.3% (11/15) at week 4, 8 and 12, respectively, in the RTX group. It remained at 72.7% (8/11) during week 4-12 in the BLM group. Excluding the data of three deceased patients, the OR rate dropped at week 24 in both groups (RTX vs BLM, 61.5% (8/13) vs 70.0% (7/10), p=1.000). At week 24, four patients with OR in both groups successfully withdrew GCs to <15 mg prednisone (RTX vs BLM, 40% (4/10) vs 66.7% (4/6), p=0.608). The serum C3 level did not significantly change, whereas the serum immunoglobulin G level significantly decreased at week 4, 8 and 12 in both groups. 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引用次数: 0
摘要
目的：免疫性血小板减少症（ITP）是一种继发于结缔组织病（CTD）的血液学表现。许多CTD-ITP患者对糖皮质激素（GCs）和免疫抑制剂（isa）难以耐受；利妥昔单抗（RTX）是推荐的二线治疗。Belimumab （BLM）显示出对CTD的疗效。我们比较了RTX和BLM的疗效和安全性。方法：收集GCs + ISAs治疗难治性CTD-ITP患者的资料。将11例接受BLM治疗的难治性CTD-ITP患者的数据与15例接受RTX治疗的患者的数据进行比较。结果：在第2周，BLM的总反应（OR）优于RTX （72.7% vs 26.7%, p=0.045）。RTX组第4、8、12周的OR率分别为60.0%（9/15）、66.7%（10/15）、73.3%（11/15）。在第4-12周，BLM组仍保持在72.7%（8/11）。排除3例死亡患者的数据，两组的OR率在第24周下降(RTX vs BLM, 61.5% （8/13) vs 70.0% (7/10), p=1.000）。在第24周，两组均有4例OR患者成功退出GCs。结论：对于GCs + ISAs难治性CTD-ITP， BLM可能是RTX安全有效的替代方案。
Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease.
Objectives: Immune thrombocytopenia (ITP) is a haematological manifestation secondary to connective tissue disease (CTD). Many patients with CTD-ITP are refractory to glucocorticoids (GCs) plus immunosuppressant agents (ISAs); rituximab (RTX) is the recommended second-line therapy. Belimumab (BLM) shows efficacy against CTD. We compared the efficacy and safety of RTX and BLM.
Methods: Data of patients with CTD-ITP refractory to GCs plus ISAs administered were collected. The data of 11 patients with refractory CTD-ITP who received BLM were compared with those of 15 patients treated with RTX.
Results: At week 2, BLM resulted in a better overall response (OR) than RTX (72.7% vs 26.7%, p=0.045). The OR rate was 60.0% (9/15), 66.7% (10/15) and 73.3% (11/15) at week 4, 8 and 12, respectively, in the RTX group. It remained at 72.7% (8/11) during week 4-12 in the BLM group. Excluding the data of three deceased patients, the OR rate dropped at week 24 in both groups (RTX vs BLM, 61.5% (8/13) vs 70.0% (7/10), p=1.000). At week 24, four patients with OR in both groups successfully withdrew GCs to <15 mg prednisone (RTX vs BLM, 40% (4/10) vs 66.7% (4/6), p=0.608). The serum C3 level did not significantly change, whereas the serum immunoglobulin G level significantly decreased at week 4, 8 and 12 in both groups. There were three patients with serious adverse effects who died of severe pneumonia during weeks 12-24.
Conclusions: BLM may be a safe and effective alternative to RTX for CTD-ITP refractory to GCs plus ISAs.
期刊介绍：
Lupus Science & Medicine is a global, peer reviewed, open access online journal that provides a central point for publication of basic, clinical, translational, and epidemiological studies of all aspects of lupus and related diseases. It is the first lupus-specific open access journal in the world and was developed in response to the need for a barrier-free forum for publication of groundbreaking studies in lupus. The journal publishes research on lupus from fields including, but not limited to: rheumatology, dermatology, nephrology, immunology, pediatrics, cardiology, hepatology, pulmonology, obstetrics and gynecology, and psychiatry.
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